On April 3, 2025 BlossomHill Therapeutics, Inc., a privately-held, clinical-stage biotechnology company focused on the design and development of small molecule medicines for treating cancer and autoimmune diseases, reported that an abstract describing the design and discovery of the company’s novel, macrocyclic, reversible, mutant-selective OMNI-EGFR inhibitor, BH-30643, was accepted for a poster presentation at the upcoming American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, IL on April 29, 2025 (Press release, BlossomHill Therapeutics, APR 3, 2025, View Source [SID1234651799]).

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"At BlossomHill, we set out to reimagine what an EGFR inhibitor could achieve as a single agent precision medicine," said Dr. Jean Cui, President and Chief Executive Officer of BlossomHill Therapeutics. "Using an intentional design approach, we targeted structural features shared across activating EGFR mutations, creating an opportunity to potently and selectively target a broad spectrum of EGFR positive lung cancers."

"The growing diversity of treatments for different subgroups of EGFR mutations has added complexity – it can be hard for a doctor or patient to know which is the right treatment," said Dr. Geoff Oxnard, Chief Medical Officer of BlossomHill Therapeutics. "We envision that a super-potent EGFR kinase inhibitor could help achieve in this disease the kinds of durable responses we are seeing with next-generation ALK and ROS1 targeted therapies."

The poster title and session information are provided below. Full abstract details, including title and text, are currently available via the AACR (Free AACR Whitepaper) online itinerary planner.

Poster title: Design and discovery of BH-30643: A novel, reversible, mutant-selective macrocyclic EGFR inhibitor invulnerable to common resistance mutations
Abstract number: 5608
Session Title: Kinase and Phosphatase Inhibitors 3, Experimental and Molecular Therapeutics
Session Date/Time: Tuesday, April 29, 2025, 2:00 p.m. – 5:00 p.m. CT
Presenting Author: Jean Cui, Ph.D., Scientific Founder, President and Chief Executive Officer, BlossomHill Therapeutics
A copy of the poster will be available on the BlossomHill website at the beginning of the AACR (Free AACR Whitepaper) poster presentation.

About BH-30643

BH-30643 is a novel, macrocyclic, reversible, mutant-selective OMNI-EGFR inhibitor for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) bearing EGFR or HER2 mutations. In preclinical studies, BH-30643 demonstrated potent antitumor activity spanning classical EGFR mutations (exon 19 deletions, L858R), atypical EGFR mutations (G719X, L861Q, S768I, etc.), and exon 20 insertions, maintaining potency in the presence of known resistance mutations. BH-30643 is currently being evaluated in the Phase 1/2 global SOLARA study (NCT06706076), which includes dose escalation followed by expansion cohorts to further evaluate BH-30643 across a range of EGFR and HER2 mutations.

On April 3, 2025 Blackstone Life Sciences and Anthos Therapeutics, Inc., a transformative, clinical-stage biopharmaceutical company developing innovative therapies for the treatment of cardiometabolic diseases, reported that Novartis has completed its acquisition of Anthos Therapeutics in a transaction valued at up to $3.1 billion (Press release, Novartis, APR 3, 2025, View Source [SID1234651798]).

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Anthos was founded by Blackstone Life Sciences and Novartis in 2019 with the exclusive global rights from Novartis to develop, manufacture, and commercialize abelacimab, a novel Factor XI inhibitor that originated at Novartis. Abelacimab is currently in Phase 3 clinical development for the prevention of stroke and systemic embolism in patients with atrial fibrillation (LILAC-TIMI 76), in addition to two phase 3 studies in patients with cancer-associated thrombosis (ASTER and MAGNOLIA). Data from these trials are expected in the second half of 2026.

Transaction Details

Anthos shareholders will receive up to $3.1 billion in total deal value, including an upfront payment of $925 million, and payments in the event certain regulatory and commercial milestones are achieved.

Advisors

Goldman Sachs & Co. LLC acted as the lead financial advisor to Anthos. Morgan Stanley & Co. LLC also served as a financial advisor, and Goodwin Procter LLP served as legal advisor to Anthos.

On April 3, 2025 Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, reported that management will participate in the 24th Annual Needham Virtual Healthcare Conference taking place on April 8, 2025 (Press release, Rocket Pharmaceuticals, APR 3, 2025, View Source [SID1234651797]). Gaurav Shah, M.D., Chief Executive Officer, will take part in a fireside chat at 8:45 a.m. ET on Tuesday, April 8.

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A webcast of the fireside chat will be available here and on the Investors section of the Company’s website. An archived replay of the webcast will be available for approximately 30 days following the event.

On April 3, 2025 Scientists at the Nano Life Science Institute (WPI-NanoLSI), Kanazawa University and colleagues reported to have developed a promising new approach to cancer treatment (Press release, Kanazawa University, APR 3, 2025, View Source [SID1234651796]). By using tiny, naturally occurring particles called extracellular vesicles (EVs), they have created a way to boost the body’s immune system to fight tumors more effectively. This breakthrough could lead to more targeted cancer therapies with fewer side effects.

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The study, published in Journal of Extracellular Vesicles, was led by Rikinari Hanayama. The research team developed a special type of engineered EVs, called antigen-presenting extracellular vesicles (AP-EVs), that activate immune cells inside tumors. These AP-EVs carry key immune-boosting signals, helping T cells—the body’s natural cancer fighters—recognize and attack tumors more effectively. This new approach enhances immune responses while reducing harmful side effects often seen in traditional cancer treatments.

Background

Many modern cancer treatments, such as immune checkpoint inhibitors and cytokine therapies, help the immune system fight cancer. However, these treatments can sometimes harm healthy tissues and cause severe side effects. Extracellular vesicles (EVs) are tiny, bubble-like structures naturally produced by cells to communicate with each other. Scientists have recently explored their potential as a targeted drug delivery system. In this study, researchers successfully modified EVs to directly deliver immune-activating molecules to tumor-fighting T cells, improving treatment effectiveness while reducing risks.

How the Research Was Conducted

The researchers tested their engineered vesicles using cell culture experiments and mouse models, analyzing their effects with imaging, flow cytometry, and molecular assays to track immune responses and tumor growth. By observing how immune cells reacted to AP-EVs, they confirmed that these vesicles could selectively stimulate T cells and enhance their tumor-fighting ability. Advanced imaging techniques also showed that AP-EVs accumulated in tumors, making them a promising tool for targeted cancer therapy.

Key Findings

Stronger Immune Response: AP-EVs helped immune cells grow and attack tumors more effectively.
Changing the Tumor Environment: The treatment made tumors more visible to the immune system, turning ‘cold’ tumors into ‘hot’ ones that are easier to attack.
Better Treatment When Combined: AP-EVs worked even better when combined with an existing immune checkpoint inhibitor (anti-PD-1 therapy).
Potential for Human Use: Researchers successfully tested human-compatible versions of AP-EVs, showing promise for future cancer therapies.
A New Approach to Cancer Treatment

Unlike traditional immunotherapy, which can cause broad immune activation and unwanted side effects, AP-EVs provide precise targeting of tumor-fighting T cells, leading to tumor eradication without harming healthy tissue or causing other unwanted side effects. AP-EVs also have the potential to improve the effectiveness of immune checkpoint inhibitors, and other T-cell therapies.

"This discovery is an important step toward using natural biological tools to improve cancer treatment," says Hanayama, lead author of the study. "By using extracellular vesicles, we can enhance immune responses with fewer side effects, potentially leading to better outcomes for patients."

Next Steps

The research team is now working on optimizing AP-EVs for clinical trials and exploring their use for other types of cancer and personalized medicine.

On April 3, 2025 Phio Pharmaceuticals Corp. (NASDAQ: PHIO) is a clinical-stage siRNA biotechnology company developing therapeutics using its proprietary INTASYL gene silencing technology to eliminate cancer, reported that podium presentations will be given on its two lead INTASYL product candidates, PH-762 and PH-894 (Press release, Phio Pharmaceuticals, APR 3, 2025, View Source [SID1234651795]).

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The ITOC conference provides a unique platform for the exchange of latest findings in immunotherapy of cancer to advance oncology drug development and delivery.

Melissa Maxwell, Phio’s Director of Research and Program Management, was awarded two speaker presentations, as follows:

Plenary Session 3: New Targets and New Leads
Date and Time: April 3, 2025, 16.30 – 16.45 PM (CEST)

Title: Targeting PD-1 with self-delivering RNAi: preclinical advances and ongoing clinical evaluation in intratumoral Immunotherapy

PH-762, an INTASYL siRNA compound targeting PD-1, is being evaluated in an ongoing clinical trial (NCT 06014086) as a neoadjuvant intratumoral therapy for cutaneous malignancies, demonstrating promising preclinical efficacy, favorable tolerability, and the potential to enhance immune responses while minimizing systemic side effects.

Plenary Session 11: What Cellular Therapies Need to Work
Date and Time: April 5, 2025, 12.15 – 12.30 PM (CEST)

Title: Silencing BRD4 to increase NK cell activity for adoptive cell therapy: a novel self- delivering RNAi approach

PH-894, an INTASYL siRNA compound, selectively silences BRD4, a key regulator of gene expression. PH-894 enhances NK cell activation and proliferation without off-target effects, presenting a promising strategy to improve adoptive cell therapy.